Myasthenic physical function scale 7 (MPFS7): self-monitoring, home-based outcome measure for people living with Myasthenia

<h3></h3> Myasthenia is the most common primary transmission disorder of the neuromuscular junction. Symptoms lead to changeable physical function, often disrupting activities of daily living, and necessitating alterations in lifestyle. Symptoms can be manged to a degree, but the fluctuating pattern is often unpredictable and inconsistent, complicating pharmacological intervention. Self-monitoring can be facilitated by appropriate outcome measures (OMs) allowing people to track their symptoms and function. Home-based OMs promote self-monitoring, facilitating recognition of changes in symptoms or ability, which can be directly attributed to disease and seek support when required. This study aimed to develop a home-based OM for use in the myasthenic population to objectively monitor symptoms and functional abilities in the community setting, independently of healthcare providers. Development consisted of a multi-step, mixed method approach: item generation and identification, data collection, item selection and refinement, home-based OM generation. Each stage involving significant input from final user group, expert panel and psychometrics (Rasch analysis), thus, concurring with FDA guidelines and best practice for OM development. The final OM was tailored to take approximately 5 minutes, to facilitate adherence and consisted of 7 items; 3 objective functional measures, and 4 patient reported items. Validation of the MPF7 is planned.